IL175749A0 - Chaperone - based therapy for niemann-pick disease - Google Patents

Chaperone - based therapy for niemann-pick disease

Info

Publication number
IL175749A0
IL175749A0 IL175749A IL17574906A IL175749A0 IL 175749 A0 IL175749 A0 IL 175749A0 IL 175749 A IL175749 A IL 175749A IL 17574906 A IL17574906 A IL 17574906A IL 175749 A0 IL175749 A0 IL 175749A0
Authority
IL
Israel
Prior art keywords
niemann
chaperone
based therapy
pick disease
pick
Prior art date
Application number
IL175749A
Original Assignee
Sinai School Medicine
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Sinai School Medicine filed Critical Sinai School Medicine
Publication of IL175749A0 publication Critical patent/IL175749A0/en

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/74—Synthetic polymeric materials
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/04—Phosphoric diester hydrolases (3.1.4)
    • C12Y301/04012—Sphingomyelin phosphodiesterase (3.1.4.12)

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Obesity (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Immunology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Endocrinology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Pulmonology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
IL175749A 2003-11-25 2006-05-18 Chaperone - based therapy for niemann-pick disease IL175749A0 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US52549703P 2003-11-25 2003-11-25
PCT/US2004/041345 WO2005051331A2 (en) 2003-11-25 2004-11-24 Chaperone-based therapy for niemann-pick disease

Publications (1)

Publication Number Publication Date
IL175749A0 true IL175749A0 (en) 2008-04-13

Family

ID=34632986

Family Applications (1)

Application Number Title Priority Date Filing Date
IL175749A IL175749A0 (en) 2003-11-25 2006-05-18 Chaperone - based therapy for niemann-pick disease

Country Status (9)

Country Link
US (1) US7750050B2 (en)
EP (1) EP1691819A4 (en)
JP (1) JP2007514661A (en)
CN (2) CN101214249A (en)
AU (1) AU2004293115A1 (en)
BR (1) BRPI0416434A (en)
CA (1) CA2546863A1 (en)
IL (1) IL175749A0 (en)
WO (1) WO2005051331A2 (en)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007089875A2 (en) * 2006-01-31 2007-08-09 Beceem Communications, Inc. Selecting modulation and coding level and spatial rate for orthogonal frequency domain modulation systems
EP2137204A4 (en) * 2007-04-13 2010-05-12 Amicus Therapeutics Inc Use of substrates as pharmacological chaperones
PL2659904T3 (en) 2008-06-26 2016-01-29 Orphazyme Aps Use of Hsp70 as a regulator of enzymatic activity
EP2289900A1 (en) 2009-08-26 2011-03-02 Humboldt Universität zu Berlin Bisphosphonates as inhibitors of acid sphingomyelinase
PT3482767T (en) 2009-08-28 2021-12-29 Icahn School Med Mount Sinai Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency
PL2646044T3 (en) 2010-11-30 2020-03-31 Orphazyme A/S Methods for increasing intracellular activity of hsp70
US10155027B2 (en) 2012-07-17 2018-12-18 Amicus Therapeutics, Inc. Alpha-galactosidase A and 1-deoxygalactonojirimycin co-formulation for the treatment of fabry disease
WO2014014938A1 (en) * 2012-07-17 2014-01-23 Amicus Therapeutics, Inc. Alpha-galactosidase a and 1-deoxygalactonojirimycin co-formulation
CA2912274A1 (en) * 2013-05-14 2014-11-20 Centogene Ag Method for the diagnosis of niemann-pick disease
EP3922242A1 (en) 2014-09-15 2021-12-15 Orphazyme A/S Arimoclomol formulation
US10898476B2 (en) 2016-04-13 2021-01-26 Orphazyme A/S Heat shock proteins and cholesterol homeostasis
EP3782624A1 (en) 2016-04-29 2021-02-24 Orphazyme A/S Arimoclomol for treating glucocerebrosidase associated disorders
UY38238A (en) * 2018-05-25 2019-12-31 Genzyme Corp PHARMACEUTICAL COMPOSITIONS FOR THE TREATMENT OF ACID SPHINGOMYELINASE DEFICIENCY
US11541105B2 (en) 2018-06-01 2023-01-03 The Research Foundation For The State University Of New York Compositions and methods for disrupting biofilm formation and maintenance
EP4247792A1 (en) 2020-11-19 2023-09-27 Zevra Denmark A/S Processes for preparing arimoclomol citrate and intermediates thereof

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3255336A (en) * 1963-02-07 1966-06-07 Hydyn Company Welding machine
US4937232A (en) 1986-09-15 1990-06-26 Duke University Inhibition of protein kinase C by long-chain bases
US4816450A (en) 1986-09-15 1989-03-28 Duke University Inhibition of protein kinase C by long-chain bases
US5244915A (en) 1990-08-31 1993-09-14 Warner-Lambert Company Amico acid derivatives cyclized at the c-terminal
US5220043A (en) 1991-03-21 1993-06-15 Ohio University Synthesis of D-erythro-sphingomyelins
US5773278A (en) * 1991-05-03 1998-06-30 Mount Sinai Medical Center Acid sphingomyelinase gene
US5227508A (en) 1992-01-24 1993-07-13 Mayo Foundation For Medical Education And Research 3-deoxy-3-substituted analogs of phosphatidylinositol
US5369030A (en) 1992-09-11 1994-11-29 Duke University Method of inducing cellular differentiations and altering cell phenotype using ceramide analogs
US6255336B1 (en) 1995-09-20 2001-07-03 The Regents Of The University Of Michigan Amino ceramide-like compounds and therapeutic methods of use
US5830916A (en) * 1996-05-23 1998-11-03 Duke University Inhibitor of ceramidase
DE69943314D1 (en) 1998-02-12 2011-05-12 Kapil N Bhalla SPHINGOLIPIDE DERIVATIVES AND METHOD FOR THEIR USE
US6218390B1 (en) 1998-12-17 2001-04-17 Synaptic Pharmaceutical Corporation Morpholinone and morpholine derivatives and uses thereof
US6756504B2 (en) 2000-04-19 2004-06-29 Yissum Research Development Company Of The Hebrew University Of Jerusalem Sphingolipids
EP1430019B1 (en) * 2001-09-26 2009-12-30 Yissum Research Development Company Of The Hebrew University Of Jerusalem Sphingolipids

Also Published As

Publication number Publication date
CA2546863A1 (en) 2005-06-09
US7750050B2 (en) 2010-07-06
WO2005051331A2 (en) 2005-06-09
CN101214249A (en) 2008-07-09
BRPI0416434A (en) 2007-02-21
AU2004293115A1 (en) 2005-06-09
WO2005051331A3 (en) 2006-03-02
US20050153934A1 (en) 2005-07-14
JP2007514661A (en) 2007-06-07
EP1691819A4 (en) 2009-05-06
EP1691819A2 (en) 2006-08-23
CN1897957A (en) 2007-01-17

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